Neurotech Pharmaceuticals Gets Priority Review for NT-501 BLA as MacTel Treatment

Neurotech Pharmaceuticals, Inc., a company pioneering advancements in sustained drug delivery for chronic retinal diseases, has announced a significant milestone. The U.S. Food and Drug Administration (FDA) has deemed the Biologic License Application (BLA) for NT-501, an investigational encapsulated cell therapy for the treatment of Macular Telangiectasia (MacTel), sufficiently complete for substantive review. The review completion target, under the Prescription Drug User Fee Act (PDUFA), is set for December 17, 2024.

MacTel is a progressive neurodegenerative retinal disease that leads to the deterioration of central vision, heavily impacting the patients' quality of life. The condition is characterized by localized degeneration of the retina with subsequent alterations to the retinal vasculature, often affecting both eyes.

NT-501 represents a novel approach for this condition. It is an ocular implant specifically designed to deliver sustained therapeutic doses of ciliary neurotrophic factor (CNTF) directly to the retina. CNTF is a neuroprotective protein known for promoting the survival and functioning of photoreceptors, which are crucial for vision. By continuously supplying CNTF, NT-501 aims to slow the degenerative process and potentially improve long-term visual outcomes for patients suffering from MacTel.

Richard Small, the Chief Executive Officer of Neurotech Pharmaceuticals, expressed significant enthusiasm regarding this development. He acknowledged the hard work of the company’s employees in reaching this important milestone, highlighting it as a notable achievement for the organization.

The core technology behind NT-501 involves the Encapsulated Cell Therapy (ECT) platform. This platform is a cell-based delivery system engineered for the long-term delivery of therapeutic proteins to treat chronic retinal diseases. The ECT system comprises a small, semi-permeable capsule containing allogeneic retinal pigment epithelial (RPE) cells. These cells are genetically modified to produce specific therapeutic proteins aimed at treating targeted diseases. The capsule is surgically inserted into the patient’s vitreous humor and attached to the sclera in an outpatient procedure. The semi-permeable membrane of the capsule allows essential nutrients to diffuse in and the therapeutic proteins to diffuse out, facilitating continuous treatment delivery directly to the retina. Additionally, the membrane serves to protect the implanted RPE cells from the patient’s immune system, ensuring their long-term functionality and survival.

Neurotech Pharmaceuticals, Inc. is a privately held, clinical-stage biotechnology firm dedicated to pioneering sustained drug delivery solutions for chronic retinal diseases. Their flagship technology, the ECT platform, is designed to enable the continuous production and delivery of therapeutic proteins to the posterior segment of the eye, addressing the needs of conditions like MacTel.